The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
This long-term, open-label study will serve as a continued access study for participants with FA who have previously participated in a vatiquinone study. The purpose of this study is to assess continued safety and efficacy of vatiquinone dosing in previously treated participants. This study addresses the medical need for participants to continue vatiquinone with a planned study duration of 3 years.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
130
Vatiquinone will be administered per dose and schedule specified in the arm.
UCLA
Los Angeles, California, United States
University of Florida
Gainesville, Florida, United States
Number of Participants With Adverse Events (AEs)
Time frame: Baseline up to 3 years
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3
Time frame: Baseline, Year 3
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University of South Florida
Tampa, Florida, United States
University of Iowa
Iowa City, Iowa, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Murdoch Children's Research Institute
Parkville, Victoria, Australia
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, Brazil
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, Canada
CHU Sainte-Justine
Montreal, Quebec, Canada
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
Paris, France
...and 4 more locations